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Blood, 1 August 2004, Vol. 104, No. 3, pp. 881-888.
Prepublished online as a Blood First Edition Paper on April 8, 2004; DOI 10.1182/blood-2003-10-3402.
 Previous Article | Table of Contents | Next Article 
TRANSPLANTATION
Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999
Charles Peters,
Lawrence R. Charnas,
Ye Tan,
Richard S. Ziegler,
Elsa G. Shapiro,
Todd DeFor,
Satkiran S. Grewal,
Paul J. Orchard,
Susan L. Abel,
Anne I. Goldman,
Norma K. C. Ramsay,
Kathryn E. Dusenbery,
Daniel J. Loes,
Lawrence A. Lockman,
Shunichi Kato,
Patrick R. Aubourg,
Hugo W. Moser, and
William Krivit
From the Department of Pediatrics, University of Minnesota, Minneapolis; the Department of Biostatistics, University of Minnesota, Minneapolis; the Department of Therapeutic Radiology, University of Minnesota, Minneapolis; Suburban Radiologic Consultants, Ltd, Minneapolis, MN; Tokai University, Isehara, Japan; Hôpital Saint-Vincent de Paul, Institut National de la Santé et de la Recherche Médicale (INSERM), Paris, France; and the Kennedy Krieger Institute, Baltimore, MD.
Cerebral X-linked adrenoleukodystrophy (X-ALD) is a disorder of very-long-chain fatty acid metabolism, adrenal insufficiency, and cerebral demyelination. Death occurs within 2 to 5 years of clinical onset without hematopoietic cell transplantation (HCT). One hundred twenty-six boys with X-ALD received HCT from 1982 to 1999. Survival, engraftment, and acute graft-versus-host disease were studied. Degree of disability associated with neurologic and neuropsychological function and cerebral demyelination were evaluated before and after HCT. Complete data were available and analyzed for 94 boys with cerebral X-ALD. The estimated 5- and 8-year survival was 56%. The leading cause of death was disease progression. Donor-derived engraftment occurred in 86% of patients. Demyelination involved parietal-occipital lobes in 90%, leading to visual and auditory processing deficits in many boys. Overall 5-year survival of 92% in patients with 0 or 1 neurologic deficits and magnetic resonance imaging (MRI) severity score less than 9 before HCT was superior to survival for all others (45%; P < .01). Baseline neurologic and neuropsychological function, degree of disability, and neuroradiologic status predicted outcomes following HCT. In this first comprehensive report of the international HCT experience for X-ALD, we conclude that boys with early-stage disease benefit from HCT, whereas boys with advanced disease may be candidates for experimental therapies.
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